This is an NDA submission presentation in class setting showing a drug submission to treat hypothyroidism.

1. Course: RGA 6201
Instructor: Dr. Anupama Gaur
Gaus Pharma
1

2. Gaus Pharma Overview
Hypothyroidism
Market Scenario
Gausroid
Regulatory plan
Post approval strategy
2

3. “Gaus” means “Foolish Person”.
US based Pharmaceutical Company
Located in Boston
700 Employees
40 Veteran Regulatory Employees.
Filing NDA for Gausroid
3

4. Deficiency of Thyroid
Hormone.
Acquired or Congenital.
2-5% World Population
3.7% in U.S Population
(~Ten million).
Women > Men
More risk in Elders.
4

5. Levothyroxine Sodium e.g.
*Synthroid(Abbott)-$520 million
*Levoxyl (Pfizer)- $15.4 million
 Problems:
-Effect on bone density
-Unstable
-Potency problem
-Costly
5

6. Levothyroxine Calcium.
Oral Replacement Therapy
New Improved Formulation:
-Restores Bone Mineral Density
-Lesser Adverse Event
-More Stable
-More Potent
-Economical
6

7. G
A
U
S
R
O
IND Application Filed in eCTD I
D
D
E
V
E
L
NDA Application Filed in eCTD O
P
M
E
N
T
Label Expansion Strategies
7

8. *CFR Title 21 part 58 (GLP)
Non
Clinical *ICH Guidelines: Safety, Quality, Multidisciplinary
studies
• Primary Pharmacodynamics
Pharmacodynamics • Secondary Pharmacodynamics
• Safety Pharmacology
Pharmacology
• Absorption,
• Distribution,
Pharmacokinetics • Metabolism
• Excretion
Single-dose toxicity
Repeat –dose toxicity
Genotoxicity
Toxicology
Carcinogenicity
Immunotoxicity
Reproductive and developmental toxicity
8

9. Gausroid CMC Complies with:
CFR Title 21 part 211 (cGMP)
ICH Guidelines
CMC Contains:
Drug Substance and Drug Product, ICH Q6A
Stability, ICH Q1A (R2)
Manufacturing Process Development
Quality, Identity and Purity ICH Q6A
9

10. Pre IND:
-Type B Meeting as per 21 CFR 312.82.
Purpose:
To Discuss Scope and Design of Phase I.
To Investigate Shortcomings.
eCTD Format through FDA’s Electronic
Submission Gateway.
10

11. Clinical Trials After IND Approval
Three phases Studies
Multi Centers Clinical Trials in US
Randomized, Double-Blinded,
Placebo Concurrent trials.
Conducted on Adults
Age 21 yrs-75 yrs.
ICH E6 guidelines: GCP
11

12. Conducted on 50 Healthy Volunteers
Purpose: To Ensure Safety
*To Evaluate Pharmacological Action.
*To Determine Side Effects
Conducted on 600 Adult Patients
Purpose: To Start Efficacy Studies and
Clinical selecting Dose for Phase III
*To Evaluate Effectiveness for Proposed
Studies Indication
On 2500 Patients with 40Effects and Risks
*Short Term Side weeks follow up
Purpose: To Ensure Efficacy.
*To Evaluate Benefit to Risk Ratio.
*To Collect additional data for Establishing
Physician Labeling.
12

13. eCTD via Electronic Submission Gateway:
-FDA Form 356h
-FDA 3397: User Fee Cover Sheet
Payment of User Fees by PDUFA IV
$1,841,500 via Wire Transfer to U.S. bank in
St. Louis.
Submission of12 copies of Final Printed
Label with NDA.
13

14. Establishment:
-Risk Evaluation and Mitigation Strategies
-RiskMAPs
 REMS Includes:
Medication Guide
Package Insert
Communication Plan for HCP’s.
14

15. Post Marketing Plans:
Clinical Studies for Secondary Indications.
Pediatric Population
Launching Dosage Strengths
Free Patients Counseling
Promotional Campaign
Committed for Safety Reporting
15

16. Thank You for Your
Attention
16

17. 17